Records containing: Muscular Atrophy, Spinal
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CLINICAL_TRIALS study record update · 2024-03-05An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-02-21Ultrasound-assisted vs Landmark Based Intrathecal Administration of Nusinersen in Adult Patients With SMA Disease: a Randomized Trial
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CLINICAL_TRIALS study record update · 2023-12-07A Multicenter, Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection When Used in Routine Medical Practice in China
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CLINICAL_TRIALS study record update · 2023-03-13Risdiplam - A Phase I, Open-Label, Multi-Period Crossover Study to Investigate the Safety, Food Effect, Bioavailability and Bioequivalence of Oral Doses of Two Different Formulations in Healthy Subjects
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CLINICAL_TRIALS study record update · 2023-03-06Natural History of SMA: Long Term Longitudinal Study and Development of a Registry
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CLINICAL_TRIALS study record update · 2022-11-17Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)
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CLINICAL_TRIALS study record update · 2022-03-23Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy Undergoing Intrathecal Treatment
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CLINICAL_TRIALS study record update · 2021-06-15A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients
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CLINICAL_TRIALS study record update · 2021-06-01A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
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CLINICAL_TRIALS study record update · 2021-02-21An Open-Label, Single-Dose, Parallel-Group, Two-Part Study to Evaluate the Pharmacokinetics and Safety of Risdiplam in Subjects With Mild or Moderate Hepatic Impairment Compared to Subjects With Normal Hepatic Function
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CLINICAL_TRIALS study record update · 2021-01-08A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2020-10-05An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2019-10-02A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients
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CLINICAL_TRIALS study record update · 2019-10-02A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients
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CLINICAL_TRIALS study record update · 2019-08-09Multicenter, Open-Label, Single-Arm Study to Evaluate Long-Term Safety, Tolerability, and Effectiveness of 10 mg/kg BID Olesoxime in Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2018-08-07International Spinal Muscular Atrophy Patient Registry
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CLINICAL_TRIALS study record update · 2016-12-22A Multicenter, Randomized, Double Blind, Placebo Controlled, Multiple Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO6885247 Following 12 Weeks of Treatment in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH).
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CLINICAL_TRIALS study record update · 2016-11-02A Single Center Study to Collect Samples From SMA Patients for Biomarker Analysis
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CLINICAL_TRIALS study record update · 2013-12-18Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?
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CLINICAL_TRIALS study record update · 2013-03-12Pilot Study of Progressive Strength Training in Spinal Muscular Atrophy